Investor Panic Sends Sarepta Down Sharply Print E-mail
By Brian Wilson   
Thursday, 10 July 2014 09:12

Earlier this morning, Sarepta Therapeutics (NASDAQ: SRPT) announced long-term outcomes (through 144 weeks) from the Phase IIb study of the drug eteplirsen for Duchenne Muscular Dystrophy. This 12 patient trial, which was initiated in February 2012, has been the primary driver of Sarepta’s valuation for roughly three years.

Although the trial met its primary endpoint, and appears to be supporting eteplirsen’s long-term efficacy against Duchenne Muscular Dystrophy, SRPT opened at about $19.00 per share (~27% lower than yesterday’s close). Investors noticed that patients in the trial have seen further declines in the 6-minute walk test relative to the latest data readout in 2013.

Keep in mind that the data do show that eteplirsen cannot “cure” muscular dystrophy, but it can slow the progress of the disease. Looking at the data below, it’s quite apparent that eteplirsen is doing its job with statistical significance.

From Sarepta’s press release:

Summary of 6MWT: Week 144 Treatment Results*

Analysis of Repeated
6MWT Values†

Baseline 6MWT (meters)

Adjusted Mean 6MWT
Change from Baseline
(meters) at 144 Weeks

Estimated Treatment
Benefit (Eteplirsen Minus


Maximum Score
Eteplirsen (n=6)





Maximum Score
Placebo/delayed-Tx (n=4)



Mean Score
Eteplirsen (n=6)





Mean Score
Placebo/delayed-Tx (n=4)



Minimum Score
Eteplirsen (n=6)





Minimum Score
Placebo/delayed-Tx (n=4)



Day 1 Score
Eteplirsen (n=6)





Day 1 Score
Placebo/delayed-Tx (n=4)




All 6MWT analyses are based on a Mixed Model Repeated Measures test.

All 6MWT analyses include the mITT population

The pre-specified primary analysis of the 6MWT results was based on the maximum score.

Long-term holders who understand the drug and its place in the world of muscular dystrophy should actually be okay with these data, because they do demonstrate that eteplirsen gives muscular dystrophy patients more time. Until a cure is invented, this drug should become the standard of care for DMD.

At time of writing, it seems that the stock is making a recovery rally after the market’s exaggerated reaction to this decent long-term trial data.

Disclosure: Long

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